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• Vol.54 No.12
• CLINICA NEUROL, 54: 1071−1073, 2014
• Vol.54 No.12 contents

Symposium 14

Exon Skipping Approach to Duchenne Muscular Dystorphy

Shin'ichi Takeda, M.D., Ph.D.¹⁾

¹⁾Translational Medical Center, National Center of Neurology and Psychiatry

Exon skipping therapy by antisense oligonucleotide is a promising approach to Duchenne muscular dystrophy (DMD). We have reported the proof-of-concept studies using morpholino on mice or dog DMD model and on patient derived cells. Based on these results, we had promoted collaborative research with a Japanese pharmaceutical company and encouraged development of DMD gene exon 53 skipping drug, then finally started an investigator-initiated clinical trial from 2013. Furthermore, we are addressing exploratory researches to expand the possibility of AON; such as, an application of AON to Fukuyama congenital muscular dystrophy, and an elucidation of AON uptake mechanism.
Full Text of this Article in Japanese PDF (464K)

(CLINICA NEUROL, 54: 1071−1073, 2014)
key words: Duchenne muscular dystrophy, dystrophin, exon skipping, antisense-oligonucleotides

(Received: 23-May-14)